公开(公告)号：US12091675B2

公开(公告)日：2024-09-17

申请号：US17296944

申请日：2019-07-15

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: C12N15/861 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755

CPC classification number: C12N15/861 ,  A61K38/37 ,  A61K48/0083 ,  C07K14/755 ,  A01K2217/075 ,  A01K2227/105 ,  A01K2267/0381

Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.

公开(公告)号：US20230023826A1

公开(公告)日：2023-01-26

申请号：US17784093

申请日：2020-12-10

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: A61K48/00 ,  A61K31/573 ,  G01N33/86 ,  A61P7/04

Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VIII polypeptide.

公开(公告)号：US20250043310A1

公开(公告)日：2025-02-06

申请号：US18797893

申请日：2024-08-08

Applicant: Takeda Pharmaceutical Company Limited

Inventor： Hanspeter Rottensteiner ,  Werner Hoellriegl

IPC: C12N15/861 ,  A61K38/37 ,  A61K48/00 ,  C07K14/755

Abstract: The present disclosure provides, among other aspects, codon-altered polynucleotides encoding Factor VIII variants for expression in mammalian cells. In some embodiments, the disclosure also provides mammalian gene therapy vectors and methods for treating hemophilia A. In some embodiments, the present disclosure provides methods for dosing a hemophilia A patient with a polynucleotide, e.g., a codon-altered polynucleotide, encoding a Factor VII polypeptide.

公开(公告)号：US11779635B2

公开(公告)日：2023-10-10

申请号：US17525759

申请日：2021-11-12

Applicant: TAKEDA PHARMACEUTICAL COMPANY LIMITED

Inventor： Alexandra Nathalie Kopic ,  Werner Hoellriegl ,  Barbara Plaimauer ,  Hanspeter Rottensteiner ,  Eva-Maria Muchitsch

IPC: A61K38/48 ,  C12N9/64 ,  A61K9/00 ,  A61K9/19

CPC classification number: A61K38/4886 ,  A61K9/0019 ,  A61K9/19 ,  C12N9/6489 ,  C12Y304/24087

Abstract: This invention relates to methods of subcutaneous administration of ADAMTS13 formulations to a treat a disease or condition associated with ADAMTS13 and VWF dysfunction. Furthermore, evidence of the unexpectedly high bioavailability of ADAMTS13 formulations administered subcutaneously is provided herein.